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Patients and Families

Welcome to the Clinical Research Support Office (CRSO) at The Children's Hospital of Philadelphia. The CRSO is a core facility providing personnel and resources to support clinical investigators. The CRSO has expertise in implementing, conducting, and monitoring clinical research and provides a variety of services from which both novice and experienced clinical researchers can benefit. The CRSO is designed to ensure the excellence and compliance of an investigator's clinical research.

Here are some frequently asked questions about clinical research studies.


What is a clinical research study?

Clinical research is medical research that involves people. Individuals can volunteer to take part in carefully conducted investigations that ultimately uncover better ways to treat, prevent, diagnose, and understand human diseases.

Clinical research can be tests of new treatments and therapies or long-term monitoring of how disease and health naturally progress. A clinical research study — also called a clinical trial or research trial — is a study of human volunteers designed to answer specific health questions. Carefully conducted research studies are the fastest and safest way to find treatments for disease and ways to improve health.

Interventional studies determine whether experimental treatments or new ways of using known therapies are safe and effective under controlled environments. Observational studies look at health issues in large groups of people or populations in natural settings.

During clinical research studies, The Children's Hospital of Philadelphia must comply with strict local, hospital, and federal guidelines. These guidelines are designed to be sure parents and children understand what they agree to do and that the possible benefits of taking part are greater than the risks. Each study has different requirements for time involved, clinic visits, treatments, tests, and hospital stays.


Who oversees clinical research at the Hospital?

Every clinical research study in the United States must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks of the study are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others who ensure that a research study is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that approves and periodically reviews the research.


Who can take part in a clinical research study?

Almost anyone can take part in a clinical research study. However, every study has specific requirements that must be met. Some requirements may include age, gender, general health, specific disease process, medical history, or medications that are currently being taken. These requirements are reviewed with the patient when inquiring about a study. If the requirements are met, then the patient has the option to take part in the study.


Why should my child or I take part in a clinical research study?

There are many reasons people choose to take part in a clinical research study. A research study may:

  • Help future patients
  • Lead to greater understanding of specific disease processes and medical advancements that could benefit others with the same condition

Can a healthy volunteer take part in a clinical research study?

Yes, a healthy person can take part in a clinical research study. There are many types of clinical research studies that require healthy volunteers. Researchers may need to compare healthy volunteers to volunteers with a specific medical condition.


What is informed consent?

Informed consent is the process of learning the key facts about a clinical study before deciding whether to take part. It is also a continuing process throughout the study designed to keep participants informed.

In an effort to help potential volunteers decide whether to take part in a study, the doctors and nurses involved in the study explain the details of the study. If the participant's native language is not English, translation assistance can be provided. The research team also provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts.

Risks and potential benefits are explained in the informed consent document. After reading the document and asking any questions, the participant decides whether to sign the document. The informed consent document is not a contract, and the participant may withdraw from the study at any time.


What are the benefits and risks of taking part in a clinical research study?

Benefits

Clinical research studies that are well-designed and well-executed provide the opportunity for eligible participants to:

  • Play an active role in his or her own healthcare
  • Participate in new research treatments before they are widely available
  • Help others by contributing to medical research

Risks

The following risks are associated with clinical research studies:

  • There may be unpleasant, serious, or even life-threatening side effects from taking part in experimental treatment
  • The experimental treatment may not be effective for the participant
  • The protocol may require more of one's time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays, or complex dosage requirements

What are side effects and adverse reactions?

Side effects are any undesired actions or effects of the experimental drug or treatment. The potential side effects are different for each study. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.


Are clinical research studies safe?

Taking part in a research study may involve inconveniences and risks. Your safety is important to the research team. The research team will discuss any possible risks with you. Taking part in a clinical research study is completely voluntary. Researchers at The Children's Hospital of Philadelphia comply with regulatory guidelines and the Hospital's Institutional Review Board. If you have any questions about any possible risks, you should talk to your study doctor or your regular doctor.


Does a participant continue to work with his or her primary healthcare provider while in a clinical research study?

Yes. Most studies provide short-term treatments related to an illness or condition but do not provide extended or complete primary healthcare. Having a participant's healthcare provider work with the research team can help ensure that other medications or treatments will not conflict with the protocol.


Can a participant leave a clinical research study after it has begun?

Yes. A participant can leave a study at any time. When withdrawing from the study, the participant should let the research team know that he or she wants to leave and the reasons for leaving.


What should people consider before taking part in a clinical research study?

People should know as much as possible about a research study before they agree to take part in it. Participants should feel comfortable asking members of the study team questions about the study, the care expected while in the study, and the cost of the study.

Participants may want to ask the following questions when talking with the study team. Some of the answers to these questions are found in the informed consent document.

  • What is the purpose of the study?
  • Who is going to be in the study?
  • Why do researchers think the experimental treatment being tested might be effective? Has it been tested before?
  • What kinds of tests and experimental treatments are involved?
  • How do the possible risks, side effects, and benefits of the study compare with my current treatment?
  • How might this study affect my daily life?
  • How long will the study last?
  • Will hospitalization be required?
  • Who will pay for the experimental treatment?
  • Will I be reimbursed for other expenses?
  • What type of long-term follow-up care is part of this study?
  • How will I know if the experimental treatment is working? Will results of the study be provided to me?
  • Who will be in charge of my care?

What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?

The study's research coordinator and doctor can answer your questions and discuss the details of the study with you. Preparing for a meeting will allow you to make sure all of your concerns are addressed. You may want to:

  • Plan ahead and write down possible questions to ask
  • Ask a friend or relative to come along for support and to hear the responses to the questions
  • Bring a tape recorder to record the discussion and replay it later

Where do the ideas for clinical research studies come from?

Ideas for clinical research studies usually come from researchers. After researchers test new therapies or procedures in the laboratory, the treatments with the most promising results are moved into clinical research studies. During a clinical research study, more information is gained about a new treatment, its risks, and how well it may or may not work.


Who sponsors clinical research studies?

The money for research studies comes from a number of funding sources, often called sponsors. These sponsors include a variety of organizations or individuals, such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies. Sponsors can also be federal agencies such as the National Institutes of Health, the Department of Defense, and the Department of Veteran's Affairs. Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.


What is a protocol?

A protocol is a study plan on which all clinical research studies are based. The plan is carefully designed to safeguard the health of the participants as well as to answer specific research questions. A protocol describes what characteristics people should have to take part in the study; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.


What is a placebo?

A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment's effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or treatment.


What is a control or control group?

A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.


What are the different types of clinical research studies?

  • Treatment trials test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
  • Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
  • Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.
  • Screening trials test the best way to detect certain diseases or health conditions.
  • Quality of life trials (or supportive care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.

What are the phases of clinical research studies?

Clinical research studies are conducted in phases. The studies in each phase have different purposes and help scientists answer different questions:

  • In Phase I trials, researchers test a new drug or treatment in a small group of people (20 to 80) for the first time. These studies evaluate safety, determine a safe dosage range, and identify side effects.
  • In Phase II trials, the study drug or treatment is given to a larger group of people (100 to 300). Phase II studies are designed to see if a drug or treatment is effective and to further evaluate its safety.
  • In Phase III trials, the study drug or treatment is given to large groups of people (1,000 to 3,000). These studies confirm effectiveness, monitor side effects, make comparisons to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
  • Phase IV trials are also known as post-marketing studies because they are done after a drug or treatment has been approved for use. These studies further examine the drug or treatment to gain additional information, including risks, benefits, and optimal use.

The above information was obtained in part from the National Library of Medicine clinicaltrials.gov Web site.